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A tailored Cln3(Q352X) mouse model for testing therapeutic interventions in CLN3 Batten disease

CLN3 Batten disease (CLN3 disease) is a pediatric lysosomal storage disorder that presents with progressive blindness, motor and cognitive decline, seizures, and premature death. CLN3 disease results from mutations in CLN3 with the most prevalent mutation, a 966 bp deletion spanning exons 7–8, affec...

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Detalles Bibliográficos
Autores principales:	Langin, Logan, Johnson, Tyler B., Kovács, Attila D., Pearce, David A., Weimer, Jill M.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7324379/ https://www.ncbi.nlm.nih.gov/pubmed/32601357 http://dx.doi.org/10.1038/s41598-020-67478-5

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